NCT06488313

A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD

A Phase 2a, Open-label, Multiple Ascending Dose Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Arcturus Therapeutics, Inc. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.

Detailed description

This a Phase 2a, open-label study of ARCT-810 in participants 12 years of age and older living with OTC deficiency. After a diet stabilization period of at least 4 weeks, all participants will be enrolled to receive ARCT-810 every two weeks, for up to five doses, at one of three dose levels. Clinic visits will occur during screening and at Days 1, 15, 29, 36, 43, 57, 60, 71, and 85. During the study, participants will remain on their current clinical management for OTC deficiency. Dose escalation or cohort expansion may occur following completion of three participants at each dose level.

Conditions

• OTC Deficiency
• Ornithine Transcarbamylase Deficiency
• OTCD

Interventions

Type	Name	Description
BIOLOGICAL	ARCT-810	ARCT-810 is an investigational medicinal product comprising Ornithine Transcarbamylase (OTC) messenger RNA (mRNA) formulated in a lipid nanoparticle (LNP).

Timeline

• Start date: 2024-11-04
• Primary completion: 2026-06-01
• Completion: 2026-09-01
• First posted: 2024-07-05
• Last updated: 2025-03-17

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study